Overview
This is a standard of care treatment guideline for allogeneic hematopoetic stem cell transplant (HSCT) in patients with primary immune deficiencies.
Description
Based on diagnosis and clinical history, a determination of the most appropriate regimen will be made based on the following prep plans:
Arm A: Fully Myeloablative Preparative Regimen, Arm B: Reduced Toxicity Ablative Preparative Regimen, Arm C: Reduced Intensity Conditioning, Arm D: No Preparative Regimen
Eligibility
Inclusion Criteria:
- Diagnosis of immunodeficiency or histiocytic disorder including the following:
- Severe combined immunodeficiency (SCID - all variants)
- Second bone marrow transplant (BMT) for SCID (after graft rejection)
- Omenn's Syndrome
- Reticular dysgenesis
- Wiskott-Aldrich syndrome
- Major histocompatibility complex (MHC) Class II deficiency (bare lymphocyte syndrome)
- Hyper IgM Syndrome (CD40 Ligand Deficiency)
- Common variable immunodeficiency (CVID) with severe phenotype
- Chronic Granulomatous Disease (CGD)
- Other severe Combined Immune Deficiencies (CID)
- Hemophagocytic Lymphohistiocytosis (HLH)
- X-linked Lymphoproliferative Disease (XLP)
- Chediak-Higashi Syndrome (CHS)
- Griscelli Syndrome
- Langerhans Cell Histiocytosis (LCH)
- Acceptable stem cell sources include:
- HLA identical or 1 antigen matched sibling donor eligible to donate bone marrow
- HLA identical or up to a 1 antigen mismatched unrelated BM donor
- Sibling donor cord blood with acceptable HLA match and cell dose as per current institutional standards
- Single unrelated umbilical cord blood unit with 0-2 antigen mismatch and minimum cell dose of >5 x 10^7 nucleated cells/kg as per current institutional guidelines
- Double unrelated umbilical cord blood units that are:
- up to 2 antigen mismatched to the patient
- up to 2 antigen mismatched to each other
- minimum cell dose of at least one single unit must be ≥ 3.5 x 10^7 nucleated cells/kg
- combined dose of both units must provide a total cell dose of ≥ 5 x 10^7 nucleated cells/kg
- Age: 0 to 50 years
- Adequate organ function and performance status.
Exclusion Criteria
- pregnant or breastfeeding
- active, uncontrolled infection and/or HIV positive
- acute hepatitis or evidence of moderate or severe portal fibrosis or cirrhosis on biopsy